Breakthrough in Cancer Treatment: Engineered Cells Inside Patients

In a pair of groundbreaking studies, scientists engineered cancer-fighting cells directly within the bodies of patients for the first time, rather than relying on traditional laboratory methods. This innovative approach was presented at the American Society of Hematology's annual meeting.

In the first trial conducted in July, four patients with multiple myeloma had their T-cells engineered in vivo with the CAR gene. These altered T-cells successfully targeted and attacked cancer cells in the patients' bone marrow.

Two of the patients appeared to be cured, with no detectable cancer cells, and a specific circulating blood protein absent. The other two patients showed signs of remission after five months. Yvonne Chen, a cancer immunotherapy researcher at UCLA, noted that the focus is now on achieving expected efficacy levels and ensuring a safe profile.

Safety concerns arose as patients experienced side effects likely due to the deactivated virus used in the process, including drops in blood pressure and mental confusion, but all recovered. In a second trial involving four patients with previously untreatable multiple myeloma, cancer cells were undetectable one month post-treatment, with one patient maintaining this status five months later.

Hematologist Joy Ho remarked that in vivo CAR-T for multiple myeloma is a promising step forward. This in vivo process, if made safer, could make CAR T-cell therapy more accessible and save lives. Currently, the lab-based CAR T-cell therapy is laborious, costly, and requires aggressive chemotherapy to prepare patients for the treatment.